Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-037, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head Control and Sitting Without External Assistance

PHILADELPHIA--(BUSINESS WIRE)-- #babies--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial in Spinal Muscular Atrophy (SMA) Type I at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland on May 8, 2024 (poster #627). EXG001-307 is a next generation recombinant adeno-associated virus (rAAV) gene therapy in development for Spinal